New platform to move potential MND drugs into clinical trials, faster

An experimental medicine platform that will enable us to more rapidly screen potential drugs in people with MND launches today on Global MND Awareness Day. 

The EXPErimental medicine Route To Success, or EXPERTS-ALS programme, will screen potential drugs more quickly, identifying those that should be tested in larger clinical trials faster. Researchers hope eligible patients can begin taking part in the study in summer 2024.

EXPERTS-ALS is led by Professor Martin Turner at the University of Oxford and UK MND RI co-director Professor Chris McDermott at the University of Sheffield. It involves 11 MND centres around the UK.

Drugs have to be tested in phase 3 clinical trials (such as the MND-SMART platform launched in 2020) to show if they will benefit patients. However, phase 3 trials are expensive and time-consuming and their success rate has been very low. This is partly because the drugs put forward for testing have often been chosen largely on data from laboratory studies, rather than from people living with MND. 

The EXPERTS-ALS programme will screen drugs in patients, looking for early signals of benefit found in blood tests. A ‘go’ or ‘no-go’ decision can be reached within a few months and successful drugs prioritised for testing in the larger phase 3 trials with a higher chance of a positive outcome. 

We are seeing a significant upsurge in drug discovery and development in the lab, which will mean more drugs to consider for clinical trials in the near future. However, we need to be much better at working out which will be the best candidates for trials. The EXPERTS-ALS platform should help the research community to increase our chances of ‘picking winners’ that will have a genuine therapeutic impact for people living with this devastating disease.

Dr Brian Dickie MBE, Director of Research at the MND Association

The National Institute for Health and Care Research (NIHR) through the Department or Health and Social Care (DHSC) has awarded £8 million to the project, which will fund the project for three and a half years. Patient charities MND Association, My Name’5 Doddie Foundation and MND Scotland, and medical research charity LifeArc intend to provide further support to extend the study to five years and support additional laboratory research.

Read more about the programme in our research blog.